Brazilian businessman Fernando Goldsztein created The Medulloblastoma initiative to speed up the journey toward brain cancer treatment for children like his son Frederico.

Helping journalists breathe context into complex issues is how @nationalpress.bsky.social makes good journalists better.

Nation Media Group investigative health + science journalist Mercy Chelangat wrote a powerful feature about Langat's family. She offered solid advice about preparing for interviews, identifying researchers and authentically connecting with rare disease patients and families

Ahrens-Nicklas and Musunuru are the scientific team behind the historic personalized CRISPR-based gene-editing therapy that saved the life of Baby K.J. Muldoon. @upenn.edu @childrensphila.bsky.social
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The @nationalpress.bsky.social Rare Disease fellows hail from 5 continents, + they'll hear a range of conversations about equity + access in the rare disease realm. We'll also highlight some of the incredible biotech advances that have improved the lives of some people living with rare disease.